Zobrazeno 1 - 10
of 48
pro vyhledávání: '"Juan J. Haro-Mora"'
Autor:
Naoya Uchida, Claire M. Drysdale, Tina Nassehi, Jackson Gamer, Morgan Yapundich, Julia DiNicola, Yoshitaka Shibata, Malikiya Hinds, Bjorg Gudmundsdottir, Juan J. Haro-Mora, Selami Demirci, John F. Tisdale
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss , Pp 121-132 (2021)
Gene editing with the CRISPR-Cas9 system could revolutionize hematopoietic stem cell (HSC)-targeted gene therapy for hereditary diseases, including sickle cell disease (SCD). Conventional delivery of editing tools by electroporation limits HSC fitnes
Externí odkaz:
https://doaj.org/article/367c1878f8f8434c82d116b980d84a7a
Autor:
Selami Demirci, Bjorg Gudmundsdottir, Quan Li, Juan J. Haro-Mora, Tina Nassehi, Claire Drysdale, Morgan Yapundich, Jackson Gamer, Fayaz Seifuddin, John F. Tisdale, Naoya Uchida
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 912-921 (2020)
Lentiviral addition of βT87Q-globin, a modified β-globin with an anti-sickling mutation, is currently being used in gene therapy trials for sickle cell disease (SCD) and β-thalassemia patients. βT87Q-globin interferes with sickle hemoglobin (HbS)
Externí odkaz:
https://doaj.org/article/b8f50309fcfd40f7925c9851843d83cf
Publikováno v:
Stem Cells Translational Medicine, Vol 9, Iss 5, Pp 590-602 (2020)
Abstract New technologies of induced pluripotent stem cells (iPSCs) and genome editing have emerged, allowing for the development of autologous transfusion therapies. We previously demonstrated definitive β‐globin production from human embryonic s
Externí odkaz:
https://doaj.org/article/9180f58806354d08a4734e42ba2dc3c7
Autor:
Alexis Leonard, Morgan Yapundich, Tina Nassehi, Jackson Gamer, Claire M. Drysdale, Juan J. Haro-Mora, Selami Demirci, Matthew M. Hsieh, Naoya Uchida, John F. Tisdale
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 430-437 (2019)
Humanized animal models are central to efforts aimed at improving hematopoietic stem cell (HSC) transplantation with or without genetic modification. Human cell engraftment is feasible in immunodeficient mice; however, high HSC doses and conditioning
Externí odkaz:
https://doaj.org/article/6db86d5a7bf54e228d1925acc5c5fa51
Autor:
Naoya Uchida, Matthew M. Hsieh, Lydia Raines, Juan J. Haro-Mora, Selami Demirci, Aylin C. Bonifacino, Allen E. Krouse, Mark E. Metzger, Robert E. Donahue, John F. Tisdale
Publikováno v:
Nature Communications, Vol 10, Iss 1, Pp 1-14 (2019)
Vectors used in gene therapy for hemoglobin disorders carry globin in a reverse-orientation to prevent the loss of key regulatory elements by RNA splicing, but this limits their efficiency. Here, the authors develop a vector carrying β-globin in a f
Externí odkaz:
https://doaj.org/article/10b9f0823d304489858931b8d82f9d3e
Autor:
Naoya Uchida, Tina Nassehi, Claire M. Drysdale, Jackson Gamer, Morgan Yapundich, Selami Demirci, Juan J. Haro-Mora, Alexis Leonard, Matthew M. Hsieh, John F. Tisdale
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 13, Iss , Pp 187-196 (2019)
Hematopoietic stem cell (HSC) gene therapy is curative for various hereditary diseases; however, high-efficiency transduction in HSCs remains crucial to improve the prospects for hemoglobinopathies. We previously optimized lentiviral transduction in
Externí odkaz:
https://doaj.org/article/bad1b67ab3294837b7e23c9ec3e25b24
Autor:
Naoya Uchida, Linhong Li, Tina Nassehi, Claire M. Drysdale, Morgan Yapundich, Jackson Gamer, Juan J. Haro-Mora, Selami Demirci, Alexis Leonard, Aylin C. Bonifacino, Allen E. Krouse, N. Seth Linde, Cornell Allen, Madhusudan V. Peshwa, Suk See De Ravin, Robert E. Donahue, Harry L. Malech, John F. Tisdale
Publikováno v:
Cell Reports Medicine, Vol 2, Iss 4, Pp 100247- (2021)
Summary: Sickle cell disease (SCD) is caused by a 20A > T mutation in the β-globin gene. Genome-editing technologies have the potential to correct the SCD mutation in hematopoietic stem cells (HSCs), producing adult hemoglobin while simultaneously e
Externí odkaz:
https://doaj.org/article/8e26d04f631d48acaf229ebbbd2bce17
Autor:
Naoya Uchida, Selami Demirci, Juan J. Haro-Mora, Atsushi Fujita, Lydia N. Raines, Matthew M. Hsieh, John F. Tisdale
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 9, Iss , Pp 247-256 (2018)
In vitro erythroid differentiation from primary human cells is valuable to develop genetic strategies for hemoglobin disorders. However, current erythroid differentiation methods are encumbered by modest transduction rates and high baseline fetal hem
Externí odkaz:
https://doaj.org/article/36b473ce1996481989274e0c3f7ae7cf
Autor:
Selami Demirci, Juan J. Haro-Mora, Alexis Leonard, Claire Drysdale, Daniela Malide, Keyvan Keyvanfar, Khaled Essawi, Raul Vizcardo, Naritaka Tamaoki, Nicholas P. Restifo, John F. Tisdale, Naoya Uchida
Publikováno v:
Stem Cell Research & Therapy, Vol 11, Iss 1, Pp 1-1 (2020)
An amendment to this paper has been published and can be accessed via the original article.
Externí odkaz:
https://doaj.org/article/07b8e6884a6c45098aefde61ecdd133f
Autor:
Juan J. Haro-Mora, Claire M. Drysdale, Malikiya Hinds, Julia DiNicola, Morgan Yapundich, Jackson Gamer, Yoshitaka Shibata, Selami Demirci, Bjorg Gudmundsdottir, John F. Tisdale, Naoya Uchida, Tina Nassehi
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss, Pp 121-132 (2021)
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss, Pp 121-132 (2021)
Gene editing with the CRISPR-Cas9 system could revolutionize hematopoietic stem cell (HSC)-targeted gene therapy for hereditary diseases, including sickle cell disease (SCD). Conventional delivery of editing tools by electroporation limits HSC fitnes